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    Summary
    EudraCT Number:2016-003705-34
    Sponsor's Protocol Code Number:RDC-PDE6A-01
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-11-26
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    A.2EudraCT number2016-003705-34
    A.3Full title of the trial
    PIGMENT – PDE6A gene therapy for retinitis pigmentosa
    PIGMENT – PDE6A Gentherapie für Retinitis Pigmentosa
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Administration of a genetically modified virus particle in patients with PDE6A-linked retinitis pigmentosa
    Verabreichung eines genetisch veränderten Viruspartikels bei Patienten mit PDE6A-bedingter Retinitis Pigmentosa
    A.4.1Sponsor's protocol code numberRDC-PDE6A-01
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorUniversitätsklinikum Tübingen
    B.1.3.4CountryGermany
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportTistou and Charlotte Kerstan Stiftung
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSTZ eyetrial am Department für Augenheilkunde
    B.5.2Functional name of contact pointDirector, Barbara Wilhelm
    B.5.3 Address:
    B.5.3.1Street AddressElfriede-Aulhorn-Straße 7
    B.5.3.2Town/ cityTübingen
    B.5.3.3Post code72076
    B.5.3.4CountryGermany
    B.5.4Telephone number4970712984898
    B.5.5Fax number49707129 5021
    B.5.6E-mailbarbara.wilhelm@stz-eyetrial.de
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namerAAV.hPDE6A vector
    D.3.4Pharmaceutical form Concentrate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOcular use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    PDE6A-linked retinitis pigmentosa
    E.1.1.1Medical condition in easily understood language
    Inherited degenerative disease of the retina
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level SOC
    E.1.2Classification code 10015919
    E.1.2Term Eye disorders
    E.1.2System Organ Class 10015919 - Eye disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLT
    E.1.2Classification code 10010464
    E.1.2Term Congenital eye disorders NEC
    E.1.2System Organ Class 10015919 - Eye disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10038914
    E.1.2Term Retinitis pigmentosa
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary aim of the trial is to test the safety of subretinal delivery of rAAV.hPDE6A at different doses in patients with PDE6A-linked retinitis pigmentosa up to day 365 after treatment.
    E.2.2Secondary objectives of the trial
    The investigation of treatment effects as reflected by patient reported outcomes and other standard clinical endpoints of visual function are secondary aims of the trial.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Study Eye & Fellow Eye; General
    • clinical diagnosis of retinitis pigmentosa
    • confirmed mutation in PDE6A gene
    • ≥ 18 years of age
    • visual acuity ≥ 20/400
    • no infection with Human Immundeficiency Virus (HIV)
    • negative pregnancy test in women with childbearing potential (a woman who is two years post-menopausal or surgically sterile is not considered to be of childbearing potential)
    • Male patients must agree to use condoms during the first 6 months post treatment.
    • Female patients of childbearing potential must agree to use an effective method of birth control during the first 6 months post treatment.
    • ability to understand and willingness to consent to study protocol
    E.4Principal exclusion criteria
    Ocular (study eye & fellow eye)
    • additional interfering ocular conditions with impact on study results (e.g. ocular opacity and advanced cataract, uveitis, amblyopia)
    • recent (6 months) ocular surgery, intravitreal or subretinal implantation of a medical device
    • disease causing mutations in another known retinitis pigmentosa gene
    • ocular infection with herpes simplex virus in medical history
    • history of ocular malignancies
    • disorders of the internal retina (e.g. retinal detachment in the patients history)
    • glaucoma defined as damage of the optic nerve
    • vascular retinal occlusion
    • diabetic patients suffering from retinopathy and/or macula edema
    • any other retinopathy due to other diseases e.g. (but not limited to) arterial hypertension, trauma or acquired inflammatory diseases (uveitis serology), contraindication to pharmacological mydriasis (e.g. history of angle block glaucoma)
    • absence of visual function on the contralateratal eye
    Systemic
    • systemic conditions (e.g. coronary heart disease, autoimmune disorders) which may affect study participation or outcome measures
    • History of poorly controlled Diabetes Mellitus type 1 or type 2
    • systemic illness or medically relevant abnormal laboratory values in blood analysis including renal and hepatic functions at inclusion
    • patients treated with oral corticoids within 14 days prior inclusion
    • current or recent participation in other study/or administration of biologic agent within the last three months
    • known sensitivity to any compound used in the study
    • contraindications to systemic immunosuppression
    • contraindications in view of the planned surgery (e.g. but not limited to anaemia Hb<10g/dl, coagulopathy with PT/PTT >1,5 fold upper limit, hypertension with values above 180 mmHg systolic and 110 mmHg diastolic) including intolerance and contraindications to general anaesthesia
    • intolerance to contrast agents used for diagnostic methods like angiography with fluoresceine or indocyanine green (e.g. but not limited to hyperthyroidism, hepatic insufficiency)
    • subject/partner of childbearing potential unwilling to use adequate contraception for four months
    • nursing or pregnant women
    • any other cause that, in the investigator‘s opinion, renders potential subjects not suitable for the study
    E.5 End points
    E.5.1Primary end point(s)
    Safety as primary endpoint will be assessed by clinical examination of ocular inflammation (slit lamp, fundus biomicroscopy, and angiography) and laboratory analyses and will be assessed by ocular and non-ocular adverse events and their relationship to the study compound. Documentation of adverse events will be in accordance by CTCAE in its current valid version.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Timepoint of evaluation of primary endpoint is D365
    E.5.2Secondary end point(s)
    1. BCVA assessed using the ETDRS visual acuity protocol
    2. Contrast sensitivity (PR Charts)
    3. Chromatic pupillography
    4. FM-28
    5. Scotopic visual field
    6. Kinetic visual field
    7. Multifocal-ERG
    8. FAF, sdOCT and angiography recordings (ICG, FLA)
    9. 6A-PRO (intervention-specific scale assessing patient reported outcome)
    10. VFQ25
    E.5.2.1Timepoint(s) of evaluation of this end point
    Assessments are performed at screening, D0, D1, D2, D14, D30, D90, D180, D365, M24, M36, M48, M60

    For further details please refer to flowchart on page 19 and 20 of the protocol.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    dose-escalation
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last follow-up visit of last subject
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 9
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 9
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state9
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Trial subjects will be controlled by regular routine examinations only or treated in subsequent studies. Currently, there is no treatment alternative available and not enough scientific data available for a compassionate use program. Close monitoring of subjects may be required in the follow-up period after the study to ensure patient safety. In any case, it is expected that subjects will continue to the treated by the investigational site.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-07-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-05-20
    P. End of Trial
    P.End of Trial StatusOngoing
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